HOME / RESOURCES / PUBLICATIONS

Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A

Thomas FP, Saporta MA, Attarian S, Sevilla T, Sivera R, Fabrizi GM, Genovese F, Gray AJ, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Paoli X, Sénéchal T, Day L, Ouyang C, Llewellyn S, Larkin M, Boutalbi Y (Journal of Clinical Neuromuscular Disease, 2022 24(1): 7–17 )

The manuscript reporting findings from our ongoing CMT&Me study has been published in the Journal of Clinical Neuromuscular Disease.

See here for more of our publications on CMT and other topics.

This manuscript is also available in the Journal of Clinical Neuromuscular Disease.

Authors

Author profiles can be found on our Who we a re page.

Laura Day

Sam Llewellyn

Mark Larkin

September 17, 2025
Publications

Copy Link

Supporting evidence-generation with expertise

See All Publications

What is the consistency of response to rimegepant at a group level in the acute treatment of migraine in UK adult patients? Protocol for a real-world, patient-centred study

O’Neil G, Abraham L, Pawinski R, Nakajima K, Bagshaw E, Fellows A, Llewellyn S, Lambru G (EHC, 2025)

Assessment of stable chronic bronchitis improvement with adjunctive long-term Mucinex® use via the cough and sputum assessment questionnaire (CASA-Q)

Divel C, Borecka O, Llewellyn S, Spangenthal S (CHEST, 2025)

Adjunctive long-term use of Mucinex® leading to improvement in stable chronic bronchitis and patient’s quality of life: A case report.

Spangenthal S, Divel C, Borecka O, Llewellyn S (Medical Reports, 2025)

Real-world use of Vitaccess Real™ platform to assess quality of life impact with long-term use of Mucinex® in stable chronic bronchitis

Spangenthal S, Divel C, Borecka O, Llewellyn S (ISOQOL, 2025)

Multiple myeloma caregiver costs and disabilities data for economic modelling and HTA submissions

Alsawady M, Tarnowska R, Kudlac A, Vincent S, Melrose D, Lied-Lied A (ISPOR Europe, 2025)

A targeted review of caregiver experiences of CAR-T therapy in ambulatory settings

Ringland C, Bagshaw E, Llewellyn S, Pugh G (ISPOR Europe, 2025)

Extrapulmonary disease burden and impact of cystic fibrosis (CF) on productivity in people with CF (pwCF) aged >12 years not treated with CF transmembrane conductance regulator modulators (CFTRm): interim analysis (IA) of the HUBBLE study

Elborn S, Mainz J, Abbott J, Carr S, Sole A, Costa S, Ganapathy V, Arteaga-Solis E, Liu J, Yuan J, Thorat T, Llewellyn S, Larkin M, De Iorio F, Kaplowitz H (ITS Annual Scientific Meeting, 2021)

Patient‑ and Caregiver‑Reported Burden of Transfusion‑Dependent β‑Thalassemia Measured Using a Digital Application

Paramore C, Levine L, Bagshaw E, Ouyang C, Kudlac A, Larkin M (Springer Nature’s, The Patient – Patient-Centered Outcomes Research, 2020)

Patient-reported burden of transfusion-dependent Beta-Thalassemia in the USA and UK measured using a digital app

Paramore C, Helfer J, Levine L, Kousoulakou H, Larkin M (ISPOR EU, 2019)

Deprivation and access to care among patients with melanoma in the UK

Quinn C, Larkin M, Jacobs I, Kandola S, Smoyer K (ISPOR EU, 2022)

Subscribe to Vitaccess

Join us and receive white papers as they are published.

Privacy Overview

This website uses cookies to improve your experience while you navigate through the website. Out of these, the cookies that are categorized as necessary are stored on your browser as they are essential for the working of basic functionalities of the website. We also use third-party cookies that help us analyze and understand how you use this website. These cookies will be stored in your browser only with your consent. You also have the option to opt-out of these cookies. But opting out of some of these cookies may affect your browsing experience.

Necessary

Always Enabled

Necessary cookies are absolutely essential for the website to function properly. These cookies ensure basic functionalities and security features of the website, anonymously.

Cookie	Duration	Description
cookielawinfo-checkbox-analytics	1 year	Set by the GDPR Cookie Consent plugin, this cookie is used to record the user consent for the cookies in the "Analytics" category .
cookielawinfo-checkbox-marketing	1 year	This cookie is set by the GDPR Cookie Consent plugin to store the user consent for the cookies in the category "Marketing".
cookielawinfo-checkbox-necessary	1 year	Set by the GDPR Cookie Consent plugin, this cookie is used to record the user consent for the cookies in the "Necessary" category .
CookieLawInfoConsent	1 year	Records the default button state of the corresponding category & the status of CCPA. It works only in coordination with the primary cookie.
JSESSIONID	session	The JSESSIONID cookie is used by New Relic to store a session identifier so that New Relic can monitor session counts for an application.
__hssrc	session	This cookie is set by Hubspot whenever it changes the session cookie. The __hssrc cookie set to 1 indicates that the user has restarted the browser, and if the cookie does not exist, it is assumed to be a new session.

Analytics

Analytical cookies are used to understand how visitors interact with the website. These cookies help provide information on metrics the number of visitors, bounce rate, traffic source, etc.

Cookie	Duration	Description
hubspotutk	5 months 27 days	HubSpot sets this cookie to keep track of the visitors to the website. This cookie is passed to HubSpot on form submission and used when deduplicating contacts.
iutk	5 months 27 days	This cookie is used by Issuu analytic system to gather information regarding visitor activity on Issuu products.
_ga	2 years	The _ga cookie, installed by Google Analytics, calculates visitor, session and campaign data and also keeps track of site usage for the site's analytics report. The cookie stores information anonymously and assigns a randomly generated number to recognize unique visitors.
_gat_gtag_UA_99254427_1	1 minute	Set by Google to distinguish users.
_ga_B2GQFRT399	2 years	This cookie is installed by Google Analytics.
_ga_JKZYG9CKXQ	2 years	This cookie is installed by Google Analytics.
_gid	1 day	Installed by Google Analytics, _gid cookie stores information on how visitors use a website, while also creating an analytics report of the website's performance. Some of the data that are collected include the number of visitors, their source, and the pages they visit anonymously.
__hstc	5 months 27 days	This is the main cookie set by Hubspot, for tracking visitors. It contains the domain, initial timestamp (first visit), last timestamp (last visit), current timestamp (this visit), and session number (increments for each subsequent session).

Marketing

Others

Other uncategorized cookies are those that are being analyzed and have not been classified into a category as yet.

Cookie	Duration	Description
AnalyticsSyncHistory	1 month	No description
li_gc	5 months 27 days	No description
ln_or	1 day	No description

Advertisement cookies are used to provide visitors with relevant ads and marketing campaigns. These cookies track visitors across websites and collect information to provide customized ads.

Cookie	Duration	Description
mc	1 year 1 month	Quantserve sets the mc cookie to anonymously track user behaviour on the website.

Functional

Functional cookies help to perform certain functionalities like sharing the content of the website on social media platforms, collect feedbacks, and other third-party features.

Cookie	Duration	Description
bcookie	1 year	LinkedIn sets this cookie from LinkedIn share buttons and ad tags to recognize browser ID.
bscookie	1 year	LinkedIn sets this cookie to store performed actions on the website.
lang	session	LinkedIn sets this cookie to remember a user's language setting.
lidc	1 day	LinkedIn sets the lidc cookie to facilitate data center selection.
UserMatchHistory	1 month	LinkedIn sets this cookie for LinkedIn Ads ID syncing.
__cf_bm	30 minutes	This cookie, set by Cloudflare, is used to support Cloudflare Bot Management.
__hssc	30 minutes	HubSpot sets this cookie to keep track of sessions and to determine if HubSpot should increment the session number and timestamps in the __hstc cookie.

Advanced patient-centric, science-driven research

Observational Studies

Qualitative Research

Registries

Health Utilities & Preference Studies

Quantitative Research

Analytics & Data Visualization

Patient-reported Outcomes

Translation & Linguistic Validation

Publication Support

Patient & Caregiver Burden Studies

Publications

Blogs

Case studies

White papers

Webinars

Study datasets

Advanced patient-centric, science-driven research

Observational Studies

Qualitative Research

Registries

Health Utilities & Preference Studies

Quantitative Research

Analytics & Data Visualization

Patient-reported Outcomes

Translation & Linguistic Validation

Publication Support

Patient & Caregiver Burden Studies

Publications

Blogs

Case studies

White papers

Webinars

Study datasets

Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A

Mark Larkin

Supporting evidence-generation with expertise

Subscribe to Vitaccess

Cookies